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J Clin Invest. 2012 May;122(5):1600-2. doi: 10.1172/JCI63066. Epub 2012 Apr 23.

Gene therapy: too much splice can spoil the dish.

Author information

1
Frontiers-in-Genetics Program and School of Life Sciences, École Polytechnique Fédérale de Lausanne, Lausanne, Switzerland. didier.trono@epfl.ch

Abstract

The use of integrating vectors for gene therapy - required for stable correction of gene expression - carries the risk of insertional mutagenesis, which can lead to activation of a tumorigenic program. In this issue of the JCI, Moiani et al. and Cesana et al. investigate how viral vectors can induce aberrant splicing, resulting in chimeric cellular-viral transcripts. The finding that this is a general phenomenon is concerning, but some of their results do suggest approaches for the development of safeguards in gene therapy vector design.

PMID:
22523063
PMCID:
PMC3336998
DOI:
10.1172/JCI63066
[Indexed for MEDLINE]
Free PMC Article

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